Multicenter Trial of Fetal Myelomeningocele Repair

描述（由申请人提供）：自1997年以来，180例胎儿在子宫内 通过开放性胎儿手术闭合脊髓脊膜膨出（MMC）。初步临床 有证据表明，这种手术减少了分流依赖性的发生率， 使小脑和脑干恢复正常 配置.然而，MMC胎儿手术的临床结果是基于 与历史对照进行比较，仅检查有效性而非安全性。 脊髓脊膜膨出修复随机试验是一项多中心非盲试验， 200名患者的随机临床试验，将在3 胎儿外科单位（FSU），加利福尼亚大学-旧金山弗朗西斯科， 费城儿童医院和范德比尔特大学医学中心， 沿着的是独立的数据和研究协调中心（DSCC） 乔治华盛顿大学生物统计中心。的主要目的 该试验是为了确定胎儿脊髓脊膜膨出的宫内修复是否在 妊娠18至25周可改善结局，如（1）死亡或 按1年生命和（2）死亡计算的需要心室减压分流 或贝利精神发育指数，与标准产后修复相比。 符合资格标准的患者将集中 随机分为子宫内或标准产后MMC修复组。 分配到胎儿手术组的患者将出院到附近的 手术后的住宿，除非不可行，在这种情况下， 在妊娠32周时返回分配的FSU，在37周时分娩 剖腹产妊娠。分配接受产后手术的患者 第37周时，该组将返回家中，返回分配的FSU， 剖腹产磁共振成像将在 入组、出院或足月妊娠，以及一岁，以确定是否 宫内修补可改善基亚里II畸形的程度。 将记录新生儿发病率以及外科手术次数 对于与MMC、肌肉力量、截肢状态和 尿液和粪便的排泄物。婴儿将在12点进行随访， 对于详细的神经运动功能分析，校正年龄为30个月， 认知测试和神经发育状态评估。此外，本发明还提供了一种方法， 对母亲的长期心理社会和生殖影响将是 评估。 总之，拟议的试验预计将证明胎儿是否 干预提供了改善的结果与合理的生活质量， 脊柱裂儿童

DESCRIPTION (provided by applicant): Since 1997, 180 fetuses have had in utero closure of myelomeningocele (MMC) by open fetal surgery. Preliminary clinical evidence suggests that this procedure reduces the incidence of shunt-dependent hydrocephalus and restores the cerebellum and brainstem to more normal configuration. However, clinical results of fetal surgery for MMC are based on comparisons with historical controls and examine only efficacy and not safety. The Myelomeningocele Repair Randomized Trial is a multi-center unblinded randomized clinical trial of 200 patients that will be conducted at three Fetal Surgery Units (FSUs), the University of California-San Francisco, Children s Hospital of Philadelphia, and Vanderbilt University Medical Center, along with an independent Data and Study Coordinating Center (DSCC), the George Washington University Biostatistics Center. The primary objective of the trial is to determine if intrauterine repair of fetal myelomeningocele at 18 to 25 weeks gestation improves outcome, as measured by (1) death or the need for ventricular decompressive shunting by one year of life and (2) death or Bayley Mental Development Index, as compared to standard postnatal repair. Consenting patients who satisfy eligibility criteria will be centrally randomized to either intrauterine or standard postnatal repair of the MMC. Patients assigned to the fetal surgery group will be discharged to nearby accommodation following surgery, unless unfeasible, in which case they will return to their assigned FSU at 32 weeks gestation for delivery at 37 weeks gestation by cesarean section. Patients assigned to the postnatal surgery group will return home and at 37 weeks, return to their assigned FSU for delivery by cesarean section. Magnetic resonance imaging will be conducted at enrollment, discharge or term gestation, and one year of age to determine if intrauterine repair improves the degree of the Chiari II malformation. Neonatal morbidity will be recorded as will the number of surgical procedures for conditions related to the MMC, muscle strength, ambulation status and urinary and fecal continence. Infants will make follow-up visits at twelve and thirty months corrected age for detailed neuromotor function analysis, cognitive testing and evaluation of neurodevelopmental status. In addition, the long term psychosocial and reproductive consequences in mothers will be evaluated. In summary, the proposed trial is expected to demonstrate whether fetal intervention offers improved outcome with a reasonable quality of life for spina bifida children.